Preprint / Version 1

An Overview of Gene Editing and Genetic Diseases

##article.authors##

  • Shiron Bagchi Westford Academy

DOI:

https://doi.org/10.58445/rars.2634

Keywords:

Gene editing, Crispr-Cas9, Talens, Down Syndrome, SCID, Non Homologous End Joining, Homology directed repair, ZFNs

Abstract

Gene editing introduces a strong potential for the future of healthcare. This paper describes what gene editing is, the differences between viral delivery systems(AAV., Lentiviruses, In-Vivo vs. Ex-Vivo), and the specific DNA repair pathways(Homology Directed Repair vs. Non-Homologous End Joining). It also dives into the many technologies that are currently being used for gene editing, like CRISPR, TALENs, and Zinc Finger Nucleases, describing the strengths/weaknesses of each technology and how each one was specifically designed to work. In addition, the paper details the cause of common genetic diseases like Down Syndrome and Severe Combined Immunodeficiency and lists potential solutions through gene editing. Ultimately, this review emphasizes the need for continued research, collaboration, and ethical oversight to responsibly integrate gene editing into modern medicine.

References

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Posted

2025-06-13